Under the guidance of our Scientific Advisory Board through a carefully managed grants process, FPWR selects research projects based on the collaborative input of researchers and parents, choosing projects that are both scientifically meritorious and highly relevant for individuals with PWS and their families.

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Evaluation of CART as a potential therapeutic target for PWS using a rat model

Funded Year: 2023

The goal of this study is to further evaluate whether the hormone, CART, is a viable therapeutic target for the treatment of the insatiable appetite associated with Prader Willi Syndrome (PWS). In this application, we propose to continue our studies through the following Aims: 1. determine if injection of CART can decrease appetite in obese...

The Role of Microglia in Prader-Willi Syndrome and a Hypothalamic Gene Therapy

Funded Year: 2023

Recently, Dr. Cao validated the safety and efficacy of a novel brain-directed gene therapy (BDNF) for metabolic and behavioral deficits observed in a preclinical mouse model of PWS, and identified BDNF as a potential therapeutic target to treat metabolic and behavioral aspects of PWS. Follow-up studies by our group investigated the genetic...

Determining beloranib’s mechanism of action to inform novel drug targets for Prader-Willi syndrome

Funded Year: 2023

Dr. Mitchell and her team have been investigating how beloranib, a drug that effectively reduced hyperphagia and induced weight loss in individuals with PWS, worked. By defining downstream effectors of beloranib’s hyperphagia-reducing action, they hope to identify a safe and effective drug to treat hyperphagia in PWS. Dr. Theresa Strong, Director...

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